Abstract
uniQure and CSL Behring agreed last year to enter their agreement, which provides CSL Behring with exclusive global rights to etranacogene dezaparvovec. CSL Behring had agreed to pay uniQure $450 million cash by May 13, 2021, and up to $1.6 billion in payments tied to achieving regulatory and commercial milestones. uniQure will also be eligible to receive tiered double-digit royalties in a range of up to a low-20s percentage of net product sales arising from the collaboration.
“This transaction positions etranacogene dezaparvovec to be made available to the largest number of hemophilia B patients as quickly as possible and provides uniQure with significant financial resources to advance and expand our pipeline of gene therapy candidates,” uniQuire CEO Matt Kapusta stated. 1
uniQure also agreed to oversee the completion of the HOPE-B pivotal trial (NCT03569891), manufacturing process validation, and the manufacturing supply of etranacogene dezaparvovec until those capabilities are transferred to CSL Behring. Clinical development and regulatory activities performed by uniQure under the agreement will be reimbursed by CSL Behring. CSL Behring will be responsible for regulatory submissions and commercialization of etranacogene dezaparvovec.
A waiting period required under the Hart-Scott-Rodino Antitrust Improvements Act of 1976 has expired, allowing the agreement to become effective.
BATTELLE, PARTNERS FORM AMPLIFYBIO WITH $200M
AmplifyBio will operate from its own 30-acre dedicated footprint, with plans to purchase an existing building and surrounding land on Battelle's West Jefferson, OH, campus. The 210,000 square-foot facility has laboratory space and equipment allowing for the company's own research and development, as well as contract research work for commercial customers.
Investors in the new company include Battelle, Viking Global Investors, Casdin Capital, and Narya Capital. Battelle will have two seats on AmplifyBio's five-person board of directors.
J. Kelly Ganjei has been appointed president and CEO of AmplifyBio, and has begun the process of finalizing his leadership team. He was previously CEO of Cognate BioServices. “Combining the strength of Battelle with our investors will uniquely position us for rapid growth and the potential to help millions of people around the world,” Ganjei said. 2
AFFINIA CLOSES $110M SERIES B
EcoR1 Capital and Farallon Capital Management coled Affinia's financing. Additional new investors included Avidity Partners, Casdin Capital, GV (formerly Google Ventures), Octagon Capital, Perceptive Advisors, RA Capital Management, TCG Crossover, and Woodline Partners LP. Existing investors Atlas Venture, F-Prime Capital, Lonza, Mass General Brigham Ventures, and New Enterprise Associates (NEA) also participated in the round.
Affinia was founded in 2019 to commercialize research from the laboratory of Luk Vandenberghe, PhD, associate professor at Massachusetts Eye and Ear and Harvard Medical School and a coinventor of AAV9. The company recently expanded its discovery laboratories in Waltham, MA, by adding process science, analytical development, and manufacturing pilot plant capabilities.
Caroline Stout, partner at EcoR1 Capital, was appointed to Affinia's board of directors.
“We are excited to partner with this syndicate of top investors to expand our platform to regulatory elements and work toward the next generation of one-time, potentially curative medicines for broader use,” Affinia CEO Rick Modi said. 3
DYNO COMPLETES $110M SERIES A
“Our CapsidMap platform brings unprecedented scale and technical sophistication to solving in vivo delivery, the key challenge for gene therapy, making therapies more effective, safe, manufacturable and capable of benefiting more patients,” said Eric Kelsic, PhD, Dyno's CEO and cofounder. 4
The company has launched collaborations totaling potentially $4 billion-plus with Novartis, Sarepta Therapeutics, and Roche and its Spark Therapeutics subsidiary.
Dyno plans to triple its workforce over the next 2 years, having doubled in size over the past 12 months to ∼50 employees. 5
Andreessen Horowitz led the $100 million Series A financing, with participation from new investors that include Casdin Capital, GV (formerly Google Ventures), Obvious Ventures, and Lux Capital—as well as founding investors Polaris Partners, CRV, and KdT Venture.
Jorge Conde, General Partner at Andreessen Horowitz, is joining Dyno's board of directors.
AVROBIO RETREATS FROM ACCELERATED APPROVAL FOR FABRY DISEASE GENE THERAPY
AVROBIO's retreat came after the FDA granted full approval of Sanofi Genzyme's Fabrazyme® (agalsidase beta) >18 years after the enzyme replacement therapy received accelerated approval based on a surrogate endpoint, reduction of GL-3 (also referred to as Gb3) inclusions in biopsied renal peritubular capillaries (PTCs). AVROBIO reasons that the surrogate endpoint could potentially apply to AVR-RD-01, whereas conversion of Fabrazyme to full approval limited the accelerated approval pathways available for new therapies to treat Fabry disease.
As a result, AVROBIO said, it intends to discuss with FDA a registration trial with a primary efficacy endpoint of clearance of GL-3/Gb3 inclusions in biopsied renal PTCs as the basis for full approval.
“We believe we have a potential new path to pursue full approval for investigational AVR-RD-01 as a first-line therapy for Fabry disease by conducting a single, head-to-head registration trial versus Fabrazyme using a kidney biopsy surrogate endpoint similar to our FAB-GT Phase II trial,” said Geoff MacKay, CEO and president of AVROBIO. 6
In the ongoing FAB-GT trial (NCT03454893), AVROBIO said it has seen 100% and 87% substrate reductions at 1 year postgene therapy in the two patients with evaluable kidney biopsies. Two additional patients had been dosed in the 2 months before May 3, with plans by AVROBIO to enroll a total of up to 14 participants in FAB-GT.
GYROSCOPE FILES, THEN POSTPONES, INITIAL PUBLIC OFFERING
“Based on the positive feedback we have received from institutional investors on the strength of our science and investigational gene therapies, we believe it's in the best interest of our existing shareholders and employees to execute our IPO in more favorable market conditions,” Gyroscope CEO Khurem Farooq said. “In the meantime, we are continuing to advance our clinical program for our investigational gene therapy, GT005, as well as our earlier stage pipeline.” 7
GT005 is Gyroscope's lead gene therapy candidate, being developed for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
In its registration statement, filed April 16, London-based Gyroscope disclosed plans to sell an unstated number of American Depositary Shares totaling $100 million.
Gyroscope said proceeds from the IPO would, in part, fund its ongoing phase I/II FOCUS clinical trial (NCT03846193), its ongoing phase II EXPLORE clinical trial (NCT04437368), and its ongoing phase II HORIZON clinical trial (NCT04566445). Gyroscope also said proceeds would be used to advance its product candidate GT011, a preclinical recombinant AAV gene therapy being developed to treat GA secondary to AMD, as well as fund general corporate purposes, including general and administrative expenses and working capital. 8
The IPO emerged 3 weeks after Gyroscope said it raised $184 million in a Series C financing led by Forbion's Growth Opportunities Fund, saying the capital would fund development of GT005.
FDA QUESTIONS LEAD TO DELAY IN PFIZER DUCHENNE MUSCULAR DYSTROPHY TRIAL
Pfizer said it is actively working with the FDA to address the questions, which include technical aspects of its potency assay matrix, so that it can begin enrolling patients in U.S. study sites. The 8-country 15-site trial is enrolling patients outside the United States.
“While we have high confidence in our current quality control overall and with a potency assay matrix which has been accepted in countries outside of the U.S., the FDA has additional technical requests that we are working to address as quickly as possible,” Pfizer Chairman and CEO Albert Bourla, DVM, PhD, told analysts on May 4 on the company's quarterly call to discuss first quarter 2021 earnings. “While we cannot speculate at this time as to when sites may open in the US, we do not expect a resolution in the first half of 2021.” 9
CIFFREO plans to enroll up to 99 boys of ages 4–7 years who have DMD and can walk independently. The trial has opened at 14 selected sites in the United Kingdom, Italy, Spain, Israel, South Korea, Japan, and Russia.